Disease Modification Strategies in Parkinson's Disease
Summary
Parkinson’s disease is characterised by progressive loss of dopaminergic neurons and accumulation of pathological protein aggregates, notably alpha‐synuclein, within the central and peripheral nervous system. Current management focuses on symptomatic relief, whereas disease modification seeks to halt or slow neurodegeneration. Approaches encompass small molecules that inhibit protein aggregation or enhance clearance pathways, immunotherapies targeting misfolded proteins, gene‐based interventions to correct or silence pathogenic mutations, and precision medicine paradigms tailored to genetic subgroups. Parallel efforts in biomarker discovery and advanced trial designs aim to identify patients at prodromal or early symptomatic stages and to establish objective measures of disease progression. Together, these strategies reflect a shift towards interventions that address underlying pathogenic mechanisms, offering the prospect of altering the natural history of the disease.
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Disease Modification Strategies in Parkinson's Disease publication trend
The graph below shows the total number of articles in disease modification strategies in parkinson's disease across all publications each year (not limited to Nature Index journals).
Technical terms
Alpha‐synuclein: A neuronal protein that aggregates into fibrils in Parkinson’s disease, contributing to neuronal dysfunction.
LRRK2 (leucine‐rich repeat kinase 2): A kinase whose mutations represent the most common genetic cause of familial Parkinson’s disease.
GBA (glucocerebrosidase): A lysosomal enzyme encoded by a gene in which mutations increase risk of Parkinson’s disease, affecting protein clearance.
Disease‐modifying therapy: An intervention designed to alter the underlying pathology or progression of a disease rather than only relieving symptoms.
Neuroprotection: Strategies aimed at preserving neuronal viability and function in the face of pathogenic insults.
References
- Quinoline Derivatives: Promising Antioxidants with Neuroprotective Potential. Antioxidants (2023).
- Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic. Movement Disorders (2018).
- Precision medicine in Parkinson’s disease: emerging treatments for genetic Parkinson’s disease. Journal of Neurology (2020).
- The Disease Modification Conundrum in Parkinson’s Disease: Failures and Hopes. Frontiers in Aging Neuroscience (2022).
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